PB2611: EVANS SYNDROME: DISEASE AWARENESS AND CLINICAL MANAGEMENT IN A NATION-WIDE ITALIAN SURVEY
Authors: Bruno Fattizzo, Valentina Carrai, Monica Crugnola, Erminia Baldacci, Marta Bellini, Costanza Bosi, Elisa Buzzatti, Domenica Caramazza, Giuseppe Carli, Monica Carpenedo, Cristina Clissa, Cristina Danesin, Maria Rosaria De Paolis, Juri Alessandro Giannotta, Vanessa Innao, Monia Marchetti, Uros Markovic, Alessandro Morotti, Mariasanta Napolitano, Andrea Patriarca, Loredana Pettine, Antonella Poloni, Elena Rivolti, Elena Rossi, Teresa Maria Santeremo, Cristina Santoro, Maria Elena Zannier, Francesco Zaja, Silvia Cantoni, Francesca Palandri, Valerio De Stefano
Journal: HemaSphere (2023)

Topic: 32. Platelet disorders Background: Evans syndrome (ES) is defined by the concomitant or subsequent association of two or more immune cytopenias, namely autoimmune hemolytic anemia (AIHA), immune thrombocytopenia (ITP), and chronic idiopathic/autoimmune neutropenia (CIN/AIN). ES management is not standardized. Aims: To assess disease awareness and clinical management of adult ES in Italy with the future aim to harmonize its diagnosis and treatment. Methods: A pre-defined structured survey including three domains 1) epidemiology, 2) diagnosis, and 3) therapy of ES (encompassing 11, 8, and 16 multiple choice questions, respectively) was administered to all Italian Centers participating in the ITP-NET initiative. The survey underwent 3 rounds of validation among 8 experts Results: From December 2022 to February 2023, 30 clinicians from 29 Italian Hospitals completed the survey. They practiced all around Italy in 23 different cities (17 university hospitals and 13 public hospitals) and followed a median of 5 (range1-45) ES patients in the last 15 years. Association of AIHA and ITP was the most reported ES type (mean 9 patients/center versus 2.8/center for AIHA+CIN or ITP+CIN, p<0.001) and 25% of patients had an underlying condition including lymphoproliferative disorders (N=16), other autoimmune diseases (15), primary immunodeficiencies (14), solid tumors (6), and stem cell transplant (5), and nearly all required treatment. Figure 1 shows the level of agreement for each diagnostic test: beyond complete blood count, direct anti-globulin test was indicated by 100%; coagulation, smear, electrophoresis and Ig dosage, ANA, ENA, anti-DNA, anti-phospholipid antibodies, and thyroid function and autoantibodies by >80%; while anti-platelets (anti-PLT) and anti-neutrophil (anti-N) autoantibodies tests were indicated by 40% of clinicians only. Regarding imaging, >60% suggested abdomen ultrasound and thorax X-ray, and only 43% CT scan. Finally, 77% indicated bone marrow evaluation at diagnosis (80% both aspirate and trephine), whilst 23% at relapse. Concerning therapy, 96% considered that ES deserves specific approach compared to isolated cytopenias, mainly for risk of relapse/complications (63%) and more complex immunopathology (76%). Only the following treatment scenarios reached >60% agreement: for ITP relapse, 60% suggested to repeat steroids if >12 months from previous cytopenia (instead of a 2nd line); for AIHA relapse, 57% suggested to differentiate treatment according to AIHA type (steroids in warm rituximab in cold), and 80% to consider recombinant erythropoietin for inadequate reticulocytosis. Regarding further therapy lines, >80% indicated that rituximab and splenectomy may have higher infectious risk if associated conditions (i.e. immunodeficiencies) or neutropenia are present; it was also suggested that TPO-RA and splenectomy (66% and 90%, respectively) may have higher thrombotic risk due to concomitant risk factors, active hemolysis, or PLT oscillations. No clear agreement was reached for anti-microbic or G-CSF prophylaxis in patients with neutropenia. Finally, >60% of clinicians advised anti-thrombotic prophylaxis in ES during severe COVID-19 infection, >70% agreed that vaccination is not contraindicated but may be deferred in case of active hemolysis/PLT<30x10^9/L. Summary/Conclusion: This survey identifies a suggested workup for adult ES, highlights high awareness regarding thrombotic and infectious complications and the importance of risk assessment to harness therapy. The heterogeneity of treatment choices among different physicians highlights the need for broader treatment harmonization.Keywords: Autoimmune hemolytic anemia (AIHA), Neutropenia, Immune thrombocytopenia (ITP)